ADial Pharmaceuticals Announces Agreement With FDA for Phase III Trial of AD04 for Alcohol Use Disorder in Patients of Targeted Genotypes

CHARLOTTESVILLE, Va., April 18, 2013 /PRNewswire/ -- ADial Pharmaceuticals, LLC ("ADial"), announced today that the U.S. Food and Drug Administration ("FDA") has agreed to allow ADial to move forward with its plan to initiate Phase III trials of AD04 as a treatment for alcohol use disorder ("AUD") in certain targeted genotypes population only. Patients will initially be screened for the targeted genotypes and only those with one or more of the targeted genotypes will be enrolled in the trial. Enrolled patients will then be randomized between the active drug and placebo. Performing the Phase III trial in only the targeted genotypes significantly reduces the trial size and associated costs and risk of the trial.

The two endpoints for demonstrating the efficacy of AD04 in the trial are percent of heavy drinking days ("PHDD") and the percent of subjects with no heavy drinking days ("PSNHDD") in the final two months of the trial. For both endpoints, a heavy drinking day is defined as a day with 4 or more drinks for women or 5 or more drinks for men. The European Medicines Authority ("EMA") has accepted PHDD as a primary endpoint for approval of drugs for the treatment of alcohol dependence and the FDA has stated that PSNHDD is an acceptable endpoint for demonstrating efficacy of AD04 in the US.

Two successful Phase III trials will be required for approval of AD04. Each trial will be 24 weeks in duration and will enroll approximately 600 patients.

"The ability to launch the Phase III trials only in subjects having the targeted genotypes represents an important breakthrough for the Company and for this promising new therapy for Alcohol Use Disorder," said William Stilley, Chief Executive Officer. "Planning is underway to commence Phase III trials later this year."

Alcohol Use Disorder (AUD)

According to the National Institute of Alcohol Abuse and Alcoholism ("NIAAA"), as many as 42 million people in the U.S. alone are afflicted by an alcohol disorder. The Centers for Disease Control ("CDC") has reported that alcohol disorders cost the U.S. economy more than $200 billion annually, with heavy drinking accounting for greater than 75% of the social and health related costs. Despite this, fewer than 5% of patients with AUD are estimated to have been diagnosed and significantly fewer receive meaningful treatment. AUD is characterized by an urge to consume alcohol and an inability to control the levels of consumption. Both acute and chronic consumption of high levels of alcohol are associated with negative social and health consequences. In the Diagnostic and Statistical Manual of Mental Disorders, 5th Edition (DSM-5) being published in May 2013, AUD replaces the disease states previously defined as alcohol abuse and alcohol dependence.

About AD04

AD04 is being developed as a treatment for alcohol use disorder in patients of select genotypes. AD04 is a serotonin-3 (5HT3) antagonist that is believed to interrupt the reinforcing effects of alcohol, and thus reduces the urge to drink. Such reinforcing effects are thought to be exaggerated in patients that have certain mutations of the genes that control serotonin function in the brain, and these are the patients targeted for treatment with AD04. AD04 contains an ultra-low dose of the active ingredient ondansetron, which is currently marketed as an anti-emetic for post-operative and post-chemotherapy nausea. Doses effective for treating alcohol addiction are well below those used to treat emesis. Clinical and non-clinical experience to date suggest that higher doses like those used for the treatment of emesis may have reduced efficacy for treating addiction and additional safety risks associated with their chronic use as would be required for treatment of addiction.

ADial has patents covering the use of ondansetron in patients with the targeted genotypes for addictions, including alcohol, obesity, and cocaine. Phase II clinical study results of AD04 for AUD have been published in the American Journal of Psychiatry and the Journal of the American Medical Association.

About ADial Pharmaceuticals, LLC

ADial Pharmaceuticals is a privately held pharmaceutical company developing medications for treating addiction diseases and disorders, including alcohol and drug addictions (cocaine, opioids, methamphetamines), as well as other addiction-like behaviors such as those related to obesity and eating disorders. The company's most advanced compound, AD04, specifically targets alcohol use disorder (previously called alcohol abuse and dependence) in patients with selected genotypes. For more information, visit http://www.adialpharma.com.